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Tarix Orphan

Tarix Orphan, LLC Announces Positive Results with TXA127 in Animal Model of Congenital Muscular Dystrophy

Results Warrant Further Research into TXA127 as a Comprehensive Single Agent Therapeutic for this Genetic Muscle Disease

Cambridge, MA (August 3, 2015): Tarix Orphan LLC, a privately held biopharmaceutical company focused on the treatment of rare neuromuscular disorders and connective tissue diseases, today announced that the company’s lead compound, TXA127, has shown positive results in the preclinical DyW model of congenital muscular dystrophy (MDC1A). Researchers showed that two doses of TXA127, a pharmaceutical formulation of the natural Ang 1‐7 peptide, remarkably reduced inflammatory and fibrotic pathology, and also induced weight/muscle gain and increased muscle function. MDC1A is a hereditary muscle disorder associated with a genetic change in the LAMA2 gene which is responsible for the production of a key muscle protein, laminin alpha‐2. MDC1A primarily affects the muscles used for movement and is one of the most common types of congenital muscular dystrophy (CMD). In addition to MDC1A’s effects on movement and motor skills, children with MDC1A may have weak respiratory muscles, leading to frequent chest infections and poor breathing at night. They may also frequently experience feeding difficulties, leading to weight loss and failure to thrive. Despite extensive research into major drivers of MDC1A, there is no cure or treatment for the condition at this time.

“We are very encouraged by the results achieved in the most commonly used mouse model of MDC1A,” said Richard Franklin, President and Chief Executive Officer of Tarix Orphan LLC. “The results seen in this study suggest that TXA127 achieves anti‐fibrotic and anti‐inflammatory activity without inhibiting growth, and may offer potential as a comprehensive single‐agent therapeutic for MDC1A. We look forward to continuing our studies with this agent in MDC1A and other neuromuscular disorders.”

About TXA127
TXA127 is a pharmaceutical grade formulation of the naturally occurring peptide Angiotensin (1‐7) which Tarix Orphan is developing for the treatment of a number of orphan and genetic diseases, with an initial focus on DMD. Additional diseases which may benefit from treatment with TXA127 include congenital muscular dystrophies, Marfan Syndrome, and amyotrophic lateral sclerosis (ALS). TXA127 is part of the “alternative renin angiotensin system (RAS)” and counteracts the “classical” RAS, which promotes hypertension, fibrosis, hypertrophy and inflammation.

About Tarix Orphan
Tarix Orphan LLC is a private biopharmaceutical company focused on the development of TXA127, a pharmaceutical formulation of the naturally occurring peptide Angiotensin (1-7), for the treatment of rare neuromuscular and connective tissue diseases. TX127 has shown therapeutic activity in animal models of Duchenne muscular dystrophy (DMD), Limb-girdle muscular dystrophy (LGMD), congenital muscular dystrophy (MDC1A), Marfan syndrome, and ALS. Tarix Orphan has broad IP protection for TXA127, and Orphan Drug Designations (ODDs) have been granted for DMD and LGMD in the United States, and for DMD in Europe. For more information on Tarix Orphan, please visit our website at www.tarixorphan.com.

Contact:
Elizabeth Wagner, Vice President
Tarix Orphan
elizabeth.wagner@tarixpharma.com

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